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Table 5 Simulation results evaluating Rule 3 when the threshold probability is set at 0.90

From: Evaluation of a multi-arm multi-stage Bayesian design for phase II drug selection trials – an example in hemato-oncology

Sample size

True benefit

Posterior mean estimate biases

Average sample sizes

% Early stopping

 

dB

dC

pA

pB

pC

A

B

C

B

C

40

−0.15

0.00

0.0092

0.0182

0.0254

39.9621

39.8136

37.9895

0.54 %

6.45 %

 

−0.05

0.00

0.0088

0.0237

0.0272

39.8054

38.8511

37.8795

3.56 %

6.90 %

 

0.00

0.00

0.0085

0.0254

0.0270

39.6095

37.9435

37.9783

6.69 %

6.53 %

 

0.05

0.00

0.0089

0.0324

0.0250

39.5103

36.5882

38.0288

11.66 %

6.37 %

 

0.10

0.00

0.0062

0.0384

0.0264

39.2740

34.3440

38.0311

19.85 %

6.37 %

 

0.15

0.00

0.0056

0.0416

0.0254

39.0452

31.9203

38.0610

29.96 %

6.22 %

 

0.20

0.00

0.0049

0.0451

0.0256

38.8153

28.6999

37.9511

42.93 %

6.61 %

 

0.25

0.00

0.0051

0.0423

0.0254

38.5798

25.0534

37.9815

57.41 %

6.60 %

 

0.30

0.00

0.0052

0.0369

0.0250

38.4489

21.2180

38.0583

71.85 %

6.33 %

 

0.35

0.00

0.0037

0.0261

0.0270

38.2338

17.3342

37.9999

83.87 %

6.49 %

 

0.40

0.00

0.0039

0.0098

0.0265

38.0706

14.1594

37.8966

92.38 %

6.79 %

 

0.45

0.00

0.0035

−0.0115

0.0266

38.0738

11.1299

37.9628

97.64 %

6.54 %

100

−0.15

0.00

0.0029

0.0093

0.0226

99.8389

99.4843

94.1617

0.55 %

6.49 %

 

−0.05

0.00

0.0031

0.0169

0.0237

99.3256

96.7377

93.7367

3.55 %

6.94 %

 

0.00

0.00

0.0020

0.0233

0.0222

99.0139

93.7839

94.1902

6.91 %

6.48 %

 

0.05

0.00

0.0013

0.0322

0.0224

98.3338

89.3276

94.1794

12.36 %

6.48 %

 

0.10

0.00

0.0012

0.0405

0.0227

97.6239

82.2664

94.1533

21.90 %

6.50 %

 

0.15

0.00

−0.0009

0.0509

0.0234

96.5788

71.2643

94.0376

37.94 %

6.60 %

 

0.20

0.00

−0.0025

0.0578

0.0235

95.7497

56.8519

94.0065

59.48 %

6.66 %

 

0.25

0.00

−0.0025

0.0577

0.0230

95.1262

41.9689

93.9749

79.99 %

6.71 %

 

0.30

0.00

−0.0030

0.0473

0.0235

94.5634

29.7273

93.9678

92.99 %

6.71 %

 

0.40

0.00

−0.0025

0.0119

0.0239

94.0066

15.1126

93.8206

99.84 %

6.79 %

 

0.45

0.00

−0.0027

−0.0110

0.0227

94.2995

11.7989

94.1851

100.00 %

6.48 %

  1. pA, pB and pC refer to the posterior means of response probability in arms A, B and C, respectively; δ*=0.15. Bold data refer to the null hypothesis of absence of any treatment difference (dB=dC=0), or to the alternative hypothesis of an expected true 0.15 benefit of treatment B (dB=0.15)